Ferrari, G;
Thrasher, AJ;
Aiuti, A;
(2021)
Gene therapy using haematopoietic stem and progenitor cells.
Nature Reviews Genetics
, 22
(4)
pp. 216-234.
10.1038/s41576-020-00298-5.
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Abstract
Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective treatment modality for monogenic disorders of the blood system such as primary immunodeficiencies and β-thalassaemia. Medicinal products based on autologous HSPCs corrected using lentiviral and gammaretroviral vectors have now been approved for clinical use, and the site-specific genome modification of HSPCs using gene editing techniques such as CRISPR–Cas9 has shown great clinical promise. Preclinical studies have shown engineered HSPCs could also be used to cross-correct non-haematopoietic cells in neurodegenerative metabolic diseases. Here, we review the most recent advances in HSPC gene therapy and discuss emerging strategies for using HSPC gene therapy for a range of diseases.
| Type: | Article |
|---|---|
| Title: | Gene therapy using haematopoietic stem and progenitor cells |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1038/s41576-020-00298-5 |
| Publisher version: | https://doi.org/10.1038/s41576-020-00298-5 |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10128384 |
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